What is taletrectinib?

Taletrectinib is an investigational targeted therapy known as a ROS1 tyrosine kinase inhibitor (TKI). It is a pill that is taken orally. Targeted therapy is a type of cancer treatment that targets proteins that control how cancer cells grow, divide, and spread.

Taletrectinib is a next-generation, potent, selective, brain penetrant, ROS1 TKI designed to overcome some of the challenges with existing ROS1 therapies. The results from both non-clinical and clinical studies showed that taletrectinib could stop the function of certain proteins that may help cancer grow. It has shown clinical activity in people who have had prior ROS1 TKI treatment and in those who have not. It has also shown clinical activity in people with ROS1-positive NSCLC who have brain metastasis.

Taletrectinib has been evaluated in two Phase 1 clinical studies in the United States (U101) and Japan (J102), which demonstrated preliminary potent and durable anti-cancer effects in participants with ROS1-positive cancers.

Taletrectinib’s efficacy and safety is being evaluated in two ongoing Phase 2 studies:

  • TRUST-I (NCT04395677) in China (active, not recruiting)
  • TRUST-II (NCT04919811), a global study with sites in North America, Europe and Asia (recruiting)

Interim results from these studies were presented at medical conferences in 2023. They demonstrated that taletrectinib shrank tumors in the body and the brain and that the cancer-inhibiting effect was sustained for durable periods of time.

Across clinical studies to date, taletrectinib has been well tolerated. Most adverse events were mild or moderate. The most common adverse events were increased liver enzymes, diarrhea, vomiting and nausea.

Taletrectinib has been granted Breakthrough Therapy Designation (BTD) in both the United States and China.

Taletrectinib has not been approved for marketing authorization in any country worldwide.

Taletrectinib is currently under review for potential marketing authorization in China. The Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) accepted New Drug Applications (NDAs) for taletrectinib for adult patients with locally advanced or metastatic ROS1-positive NSCLC who either have or have not previously been treated with ROS1 TKIs.